LT3265568T - Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene - Google Patents
Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 geneInfo
- Publication number
- LT3265568T LT3265568T LTEP16762307.3T LT16762307T LT3265568T LT 3265568 T LT3265568 T LT 3265568T LT 16762307 T LT16762307 T LT 16762307T LT 3265568 T LT3265568 T LT 3265568T
- Authority
- LT
- Lithuania
- Prior art keywords
- gene
- mutations
- prpf31
- retinal degeneration
- degeneration caused
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Ophthalmology & Optometry (AREA)
- Mycology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562129638P | 2015-03-06 | 2015-03-06 | |
| US201562147307P | 2015-04-14 | 2015-04-14 | |
| PCT/US2016/021226 WO2016144892A1 (en) | 2015-03-06 | 2016-03-07 | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| LT3265568T true LT3265568T (en) | 2020-08-25 |
Family
ID=56879585
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| LTEP16762307.3T LT3265568T (en) | 2015-03-06 | 2016-03-07 | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US20180043034A1 (en) |
| EP (2) | EP3748007B1 (en) |
| JP (3) | JP6812368B2 (en) |
| CY (1) | CY1123158T1 (en) |
| DK (1) | DK3265568T3 (en) |
| ES (1) | ES2806054T3 (en) |
| HK (1) | HK1247637A1 (en) |
| HR (1) | HRP20201225T1 (en) |
| HU (1) | HUE051491T2 (en) |
| LT (1) | LT3265568T (en) |
| PL (1) | PL3265568T3 (en) |
| PT (1) | PT3265568T (en) |
| RS (1) | RS60668B1 (en) |
| SI (1) | SI3265568T1 (en) |
| WO (1) | WO2016144892A1 (en) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| KR20220056884A (en) | 2011-04-22 | 2022-05-06 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | Adeno-associated virus virions with variant capsid and methods of use thereof |
| ES2897508T3 (en) | 2013-05-31 | 2022-03-01 | Univ California | Adeno-associated virus variants and methods of using them |
| SG10201810150UA (en) | 2014-03-17 | 2018-12-28 | Adverum Biotechnologies Inc | Compositions and methods for enhanced gene expression in cone cells |
| WO2016141078A1 (en) | 2015-03-02 | 2016-09-09 | Avalanche Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| US20180043034A1 (en) * | 2015-03-06 | 2018-02-15 | Massachusetts Eye And Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| WO2016154344A1 (en) * | 2015-03-24 | 2016-09-29 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| CN116286986A (en) | 2016-07-29 | 2023-06-23 | 加利福尼亚大学董事会 | Adeno-associated virus virions with variant capsids and methods of use thereof |
| CN110225975A (en) | 2016-08-23 | 2019-09-10 | 阿库斯股份有限公司 | For treating the composition and method of non-age-dependent hearing impairment in people experimenter |
| WO2018075798A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| JP7330899B2 (en) | 2017-05-10 | 2023-08-22 | マサチューセッツ アイ アンド イヤー インファーマリー | Methods and compositions for altering viral assembly-activating protein (AAP) dependence |
| CN110709511A (en) | 2017-08-28 | 2020-01-17 | 加利福尼亚大学董事会 | Adeno-associated virus capsid variants and methods of use thereof |
| AU2019276645A1 (en) * | 2018-06-01 | 2020-11-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of dominant retinitis pigmentosa |
| WO2020102740A2 (en) * | 2018-11-16 | 2020-05-22 | Spark Therapeutics, Inc. | Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins |
| WO2021168362A1 (en) | 2020-02-21 | 2021-08-26 | Akouos, Inc. | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| WO2024086352A2 (en) * | 2022-10-21 | 2024-04-25 | Biogen Ma Inc. | Compositions and methods for treating retinitis pigmentosa |
| WO2024123461A1 (en) * | 2022-12-05 | 2024-06-13 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Pigment epithelium-derived factor peptides and use for treating retinal degeneration |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE3023787A1 (en) | 1980-06-25 | 1982-01-21 | Studiengesellschaft Kohle mbH, 4330 Mülheim | METHOD FOR INCREASING THE INCORPORATION AND EXPRESSION OF GENETIC MATERIAL IN THE CORE OF INTACT CELLS BY LIPOSOME |
| US4980286A (en) | 1985-07-05 | 1990-12-25 | Whitehead Institute For Biomedical Research | In vivo introduction and expression of foreign genetic material in epithelial cells |
| ATE68013T1 (en) | 1985-07-05 | 1991-10-15 | Whitehead Biomedical Inst | EXPRESSION OF FOREIGN GENETIC MATERIAL IN EPITHELIAL CELLS. |
| ATE117375T1 (en) | 1987-09-11 | 1995-02-15 | Whitehead Biomedical Inst | TRANSDUCTION ALTERED FIBROBLASS AND THEIR APPLICATION. |
| DE3851153T2 (en) | 1987-12-11 | 1995-01-05 | Hughes Howard Med Inst | GENETIC MODIFICATION OF ENDOTHELIAL CELLS. |
| DE68927996T2 (en) | 1988-02-05 | 1997-12-04 | Hughes Howard Med Inst | MODIFIED HEPATOCYTES AND THEIR USE |
| DK0452457T3 (en) | 1989-11-03 | 1998-03-02 | Univ Vanderbilt | Method for in vivo removal of functional foreign genes |
| ATE163046T1 (en) | 1990-10-31 | 1998-02-15 | Somatix Therapy Corp | GENETIC MODIFICATION OF ENDOTHELIAL CELLS |
| ES2150832B1 (en) | 1996-06-12 | 2001-06-16 | Fichtel & Sachs Ag | OPERATING DEVICE FOR THE OPERATION, IN PARTICULAR PNEUMATIC OPERATION, OF A FRICTION CLUTCH. |
| US6569662B1 (en) * | 2000-01-21 | 2003-05-27 | Hyseq, Inc. | Nucleic acids and polypeptides |
| EP1381276A4 (en) * | 2001-04-13 | 2005-02-02 | Univ Pennsylvania | Method of treating or retarding the development of blindness |
| US20040208847A1 (en) * | 2003-03-28 | 2004-10-21 | Fabienne Rolling | Method and vectors for selectively transducing retinal pigment epithelium cells |
| US8211634B2 (en) * | 2006-02-14 | 2012-07-03 | Dana-Farber Cancer Institute, Inc. | Compositions, kits, and methods for identification, assessment, prevention, and therapy of cancer |
| CA2683469C (en) * | 2007-04-12 | 2019-11-26 | The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
| US20090011040A1 (en) * | 2007-05-02 | 2009-01-08 | Naash Muna I | Use of compacted nucleic acid nanoparticles in non-viral treatments of ocular diseases |
| WO2009134681A2 (en) * | 2008-04-30 | 2009-11-05 | The Trustees Of The University Of Pennsylvania | Aav7 viral vectors for targeted delivery of rpe cells |
| EP2320937A4 (en) * | 2008-06-30 | 2013-01-16 | Univ Johns Hopkins | Compositions and methods for the treatment of ocular oxidative stress and retinitis pigmentosa |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| TWI775096B (en) * | 2012-05-15 | 2022-08-21 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | Treatment of amd using aav sflt-1 |
| CN105120901A (en) * | 2012-07-11 | 2015-12-02 | 宾夕法尼亚大学托管会 | AAV-mediated gene therapy for RPGR X-linked retinal degeneration |
| ES2857751T3 (en) | 2013-10-11 | 2021-09-29 | Massachusetts Eye & Ear Infirmary | Methods for predicting ancestral virus sequences and their uses |
| EP3154573A1 (en) * | 2014-06-13 | 2017-04-19 | Universidade De Santiago De Compostela | Nanoparticulate systems for use in gene transfer or gene delivery |
| US20180043034A1 (en) * | 2015-03-06 | 2018-02-15 | Massachusetts Eye And Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
-
2016
- 2016-03-07 US US15/555,915 patent/US20180043034A1/en not_active Abandoned
- 2016-03-07 EP EP20171646.1A patent/EP3748007B1/en active Active
- 2016-03-07 ES ES16762307T patent/ES2806054T3/en active Active
- 2016-03-07 RS RS20200945A patent/RS60668B1/en unknown
- 2016-03-07 HU HUE16762307A patent/HUE051491T2/en unknown
- 2016-03-07 WO PCT/US2016/021226 patent/WO2016144892A1/en active Application Filing
- 2016-03-07 PT PT167623073T patent/PT3265568T/en unknown
- 2016-03-07 DK DK16762307.3T patent/DK3265568T3/en active
- 2016-03-07 JP JP2017564778A patent/JP6812368B2/en active Active
- 2016-03-07 SI SI201630873T patent/SI3265568T1/en unknown
- 2016-03-07 LT LTEP16762307.3T patent/LT3265568T/en unknown
- 2016-03-07 PL PL16762307T patent/PL3265568T3/en unknown
- 2016-03-07 EP EP16762307.3A patent/EP3265568B1/en active Active
-
2018
- 2018-05-25 HK HK18106900.6A patent/HK1247637A1/en unknown
-
2020
- 2020-08-04 CY CY20201100718T patent/CY1123158T1/en unknown
- 2020-08-04 HR HRP20201225TT patent/HRP20201225T1/en unknown
- 2020-08-13 US US16/992,689 patent/US20210069347A1/en active Pending
- 2020-12-16 JP JP2020208068A patent/JP7239550B2/en active Active
-
2023
- 2023-03-02 JP JP2023031931A patent/JP2023071867A/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3265568A1 (en) | 2018-01-10 |
| JP2021059561A (en) | 2021-04-15 |
| SI3265568T1 (en) | 2020-10-30 |
| CY1123158T1 (en) | 2021-10-29 |
| JP7239550B2 (en) | 2023-03-14 |
| JP2023071867A (en) | 2023-05-23 |
| HUE051491T2 (en) | 2021-03-01 |
| JP2018511652A (en) | 2018-04-26 |
| DK3265568T3 (en) | 2020-08-10 |
| HK1247637A1 (en) | 2018-09-28 |
| US20210069347A1 (en) | 2021-03-11 |
| EP3265568A4 (en) | 2018-08-08 |
| EP3265568B1 (en) | 2020-05-06 |
| WO2016144892A1 (en) | 2016-09-15 |
| JP6812368B2 (en) | 2021-01-13 |
| HRP20201225T1 (en) | 2020-11-13 |
| RS60668B1 (en) | 2020-09-30 |
| PT3265568T (en) | 2020-08-20 |
| EP3748007B1 (en) | 2024-06-26 |
| US20180043034A1 (en) | 2018-02-15 |
| EP3748007A1 (en) | 2020-12-09 |
| ES2806054T3 (en) | 2021-02-16 |
| PL3265568T3 (en) | 2020-11-30 |
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